Josh Mandel-Brehm, Camp4 Therapeutics CEO (Camp4)

Go­ing af­ter the phe­nom­e­non of 're­gR­NA,' Rick Young's Camp4 se­cures a launch round to push two pro­grams for­ward

You’ve heard of mR­NA, large­ly thanks to the Covid-19 vac­cines de­vel­oped by Pfiz­er/BioN­Tech and Mod­er­na. If you watch the R&D space close­ly, you’ve al­so prob­a­bly heard of oR­NA and eR­NA.

Rick Young

Now, a new biotech out of Rick Young’s White­head In­sti­tute lab at MIT wants to in­tro­duce you to reg­u­la­to­ry RNA, or re­gR­NA.

The com­pa­ny is Camp4 Ther­a­peu­tics, and it’s locked down its first fundraise with a $45 mil­lion Se­ries A on Tues­day. Led by CEO Josh Man­del-Brehm, Camp4 wants to cre­ate oligonu­cleotide-based med­i­cines that con­trol pro­tein ex­pres­sion to help reg­u­late any kind of ge­net­ic dis­ease.

It’s an ef­fort that got start­ed back in 2016, Man­del-Brehm told End­points News, when Young teamed up with sci­en­tif­ic co-founder Leonard Zon at Boston Chil­dren’s Hos­pi­tal. The core of their re­search cen­tered on un­der­stand­ing how cells make de­ci­sions to con­trol their genes, and they be­gan map­ping out cells them­selves.

To do so re­quired hu­man cells, a “whole ar­ray” of next-gen­er­a­tion se­quenc­ing tech­niques and a part­ner to ap­ply a steady AI hand in gen­er­at­ing da­ta, Man­del-Brehm said. Young and Zon be­came able to con­trol drug sig­nal­ing for non-can­cer in­di­ca­tions where they could change a dis­ease’s un­der­ly­ing gene ex­pres­sion in or­der to spur a ther­a­peu­tic ben­e­fit.

They ran in­to a prob­lem fo­cus­ing orig­i­nal­ly on small mol­e­cules — the drugs worked too well, the CEO told End­points, al­ter­ing the genes to a de­gree much more than they would have liked.

“They were ac­ti­vat­ing too many genes. It was too non­spe­cif­ic for us,” Man­del-Brehm said. “It was a good prob­lem to have, but we asked our­selves, ‘How do we get more spe­cif­ic?’”

Young then reeval­u­at­ed the way he thought about tran­scrip­tion, lead­ing him to RNA re­search. While look­ing in­to the mat­ter, he turned to a class of RNA mol­e­cules that don’t con­tain a core pro­tein and are on­ly tran­scribed. These RNAs had been known to re­searchers be­fore, but few knew of their func­tion be­cause they’d get se­quenced out with rel­a­tive fre­quen­cy.

Rather than con­tin­ue ig­nor­ing these reg­u­la­to­ry RNAs, the founders be­gan try­ing to har­ness them for drug­gable pur­pos­es. The cel­lu­lar maps they’d cre­at­ed for the ear­li­er ef­fort proved piv­otal here, Man­del-Brehm said, be­cause re­searchers can’t iden­ti­fy or char­ac­ter­ize the re­gR­NAs with­out them.

All of a sud­den, Camp4’s plat­form shift­ed to tar­gets. Young and Zon knew they were on­to some­thing and had one last puz­zle piece to solve: how to best at­tack the dis­eases they want­ed to treat, giv­en that small mol­e­cules didn’t suit them. They set­tled on oligonu­cleotides due to the vast amount of pre­vi­ous re­search that’s gone in­to the field.

Over the last 15 months, Camp4 has built out its pre­clin­i­cal pipeline and IP, but they need­ed cash to take things fur­ther. They got a boost in ear­ly 2020 when they signed a li­cens­ing deal with Bio­gen to col­lab­o­rate on mi­croglial cells re­lat­ed to the CNS. But for their own pipeline, the Cam­bridge, MA-based biotech is aim­ing to ini­tial­ly tar­get dis­eases for the liv­er and CNS, hop­ing to file INDs by the end of next year for pro­grams tar­get­ing OTC de­fi­cien­cy and Dravet syn­drome.

Man­del-Brehm has big­ger plans than that though, as he con­tin­ues chart­ing Camp4’s path for­ward. The biotech has about 25 to 30 CNS dis­or­ders they’re look­ing at, and the CEO is aim­ing to be at the fore­front of this new area of bi­ol­o­gy.

“The punch­line is no one is able to go af­ter up­reg­u­la­tion dis­eases, but we’re tak­ing ad­van­tage,” he said. “Peo­ple have al­ways thought of down­reg­u­la­tion, but up is a whole dif­fer­ent cat­e­go­ry. Our sense is it’s go­ing to be a very hot field.”

5AM Ven­tures and North­pond Ven­tures led Tues­day’s fi­nanc­ing along­side ex­ist­ing in­vestors An­dreessen Horowitz, Po­laris Part­ners and The Kraft Group.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

Jeffrey Bluestone, Sonoma CEO (Photo credit: Steve Babuljak)

Jeff Blue­stone just raised $265M to de­vel­op cu­ra­tive cell ther­a­pies. We asked him how

Jeff Bluestone had some big goals in mind when he decided to make a switch from a decades-long career in academia and non-profit research to a biotech startup CEO. And now — 18 months after the $40 million launch party — he has a whole lot more money on hand to pay for the considerable amount of work ahead at Sonoma Biotherapeutics.

This morning Bluestone is taking the wraps off a $265 million B round after boosting the core syndicate of A-list investors he started with. Even by today’s standards, that sum dwarfs the kind of $100 million-plus megarounds that have become standard fare in biotech over the last 2 years.

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Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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